Efficacy and Safety of M281 in Adults With Warm Autoimmune Hemolytic Anemia

NCT04119050 | Active Not Recruiting Phase 2 DMC FDA Drug

• 4 other identifiers: CR108987MOM-M281-0062019-000720-172023-505321-14-00
• Study Type: interventional
• Target: 118
• Locations: 19 countries
• Sites: 171

Brief Summary

The main purpose of this study is to evaluate the efficacy and safety of M281 in participants with warm autoimmune hemolytic anemia (wAIHA).

Conditions

Warm Autoimmune Hemolytic Anemia

Keywords

Warm Autoimmune Hemolytic Anemia; M281 (Nipocalimab); wAIHA; JNJ-80202135

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants Achieving Durable Response of Improvement in Hemoglobin (Hgb)

  Up to Week 20 of the double-blind period

Secondary Outcomes (22)

• Change From Baseline in the Total Score From the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale at the Time of Durable Response

  Baseline (Day 1, Week 0) through Week 24

• Change From Baseline in the Total Score From the FACIT-Fatigue Scale at the end of the Double-blind Period (Week 24)

  Baseline (Day 1, Week 0) through Week 24 of the double-blind period

• Change from Baseline in Average Daily Dose of Prednisone or Equivalent

  Baseline (Day 1, Week 0) and at Week 24

• Number of Participants That Simultaneously Attain Normal Lactate Dehydrogenase, Haptoglobin, and Indirect Bilirubin Levels at a Minimum of 3 Consecutive Visits After Baseline

  Baseline (Day 1, Week 0) through Week 24

• Percentage of Participants who Experience at Least a 2 g/dL Increase in Hgb From Baseline and Normalization of Lactate Dehydrogenase, Haptoglobin, and Indirect Bilirubin at any Time During the Study

  Baseline (Day 1, Week 0) through Week 24

Study Arms (5)

M281 administered every 4 weeks (double-blind period)

EXPERIMENTAL

Participants will receive M281 administered every 4 weeks alternating with placebo every 4 weeks during the 24 weeks double-blind period.

Drug: M281; Drug: Placebo

M281 administered every 2 weeks (double-blind period)

EXPERIMENTAL

Participants will receive M281 administered every 2 weeks during the 24 weeks double-blind period.

Drug: M281

Placebo administered every 2 weeks (double-blind period)

EXPERIMENTAL

Participants will receive M281 matching placebo administered every 2 weeks during the 24 weeks double-blind period.

Drug: Placebo

M281 administered every 4 weeks (open-label extension period)

EXPERIMENTAL

Participants will receive M281 administered every 4 weeks during the 144 weeks open-label extension period.

Drug: M281

M281 administered every 2 weeks (open-label extension period)

EXPERIMENTAL

Participants will receive M281 administered every 2 weeks during the 144 weeks open-label extension period.

Drug: M281

Interventions

M281 DRUG

M281 injection administered as intravenous infusion

Also known as: Nipocalimab, JNJ-80202135

M281 administered every 2 weeks (double-blind period); M281 administered every 2 weeks (open-label extension period); M281 administered every 4 weeks (double-blind period); M281 administered every 4 weeks (open-label extension period)

Placebo DRUG

Placebo administered as intravenous infusion

M281 administered every 4 weeks (double-blind period); Placebo administered every 2 weeks (double-blind period)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants greater than or equal to (\>=)18 years of age
• Have been diagnosed with warm autoimmune hemolytic anemia (wAIHA) for at least 3 months, and are currently receiving treatment for wAIHA or have previously received treatment for wAIHA (treatment-naive participants are not eligible)
• Participants must be able to understand and voluntarily provide written informed consent to participate in the study and comply with all study procedures

You may not qualify if:

• Participants must not be pregnant or breastfeeding
• Participants must not have other clinically relevant abnormalities currently or in their history that the Investigator would deem them ineligible to participate
• Have been diagnosed with cold antibody autoimmune hemolytic anemia (AIHA), cold agglutinin syndrome, mixed type (that is, warm and cold) AIHA, or paroxysmal cold hemoglobinuria

Sponsors & Collaborators

• Janssen Research & Development, LLC: lead

Study Sites (171)

MemorialCare Medical Group

Fountain Valley, California, 92708, United States

Location

University of Southern California

Los Angeles, California, 90033, United States

Location

Compassionate Cancer Care

Riverside, California, 92501, United States

Location

American Institute of Research

Whittier, California, 90603, United States

Location

University of Colorado Anschutz Medical Campus

Aurora, Colorado, 80045, United States

Location

GNP Research

Cooper City, Florida, 33024, United States

Location

University of Florida College of Medicine

Gainesville, Florida, 32610, United States

Location

21st Century Oncology

Jacksonville, Florida, 32204, United States

Location

Lakes Research

Miami Lakes, Florida, 33014, United States

Location

AdventHealth Cancer Institute

Orlando, Florida, 32804, United States

Location

Children's Research Institute

St. Petersburg, Florida, 33701, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Alliance for Multispeciality Research

Merriam, Kansas, 66204, United States

Location

Tulane University School of Medicine

New Orleans, Louisiana, 70112, United States

Location

Henry Ford Medical Center

Detroit, Michigan, 48202, United States

Location

Research Medical Center

Kansas City, Missouri, 64132, United States

Location

Montefiore Medical Center

Lake Success, New York, 11042, United States

Location

Monter Cancer Center

Lake Success, New York, 11042, United States

Location

Hematology Oncology Associates of Rockland

Nyack, New York, 10960, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

East Carolina University

Greenville, North Carolina, 27834, United States

Location

Taussig Cancer Insititute Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

The Ohio State University- James Cancer Hospital

Columbus, Ohio, 43210, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

West Penn Hospital

Pittsburgh, Pennsylvania, 15224, United States

Location

University of Utah

Salt Lake City, Utah, 84132, United States

Location

Northwest Medical Specialists

Tacoma, Washington, 98405, United States

Location

University of Wisconsin Carbone Cancer Center

Madison, Wisconsin, 53792, United States

Location

Hospital das Clínicas da Faculdade de Medicina de Botucatu

Botucatu, 18610-070, Brazil

Location

Impar Servicos Hospitalares S/A - Hospital Brasilia

Brasília, 71681-603, Brazil

Location

CTO Centro de Tratamento Oncologico Unidade Belem

Campinas, 13083-887, Brazil

Location

Hospital Das Clinicas Da Universidade Federal De Goias

Goiânia, 74605-020, Brazil

Location

Complexo Hospitalar de Niteroi

Niterói, 24020-096, Brazil

Location

Oncoclinicas - Unidade OC Oncoclinicas Multihemo Ilha do Leite

Recife, 50040-000, Brazil

Location

Nucleo de Oncologia da Bahia

Salvador, 40170-110, Brazil

Location

Hospital Sao Rafael

Salvador, 41253 190, Brazil

Location

CEPHO Centro de Estudos e Pesquisa de Hematologia e Oncologia

Santo André, 09060 650, Brazil

Location

Hospital Santa Marcelina

Santo André, 09060-870, Brazil

Location

Fundacao Faculdade Regional de Medicina de Sao Jose do Rio Preto Hospital de Base

São José do Rio Preto, 15090 000, Brazil

Location

Sociedade Beneficente de Senhoras Hospital Sirio Libanes

São Paulo, 01308 050, Brazil

Location

Universidade Federal de Sao Paulo

São Paulo, 04024-002, Brazil

Location

Hospital Das Clinicas Da Faculdade De Medicina Da USP

São Paulo, 05403 000, Brazil

Location

Beijing Chao-yang Hospital, Capital Medical University

Beijing, 100020, China

Location

Peking University People's Hospital

Beijing, 100044, China

Location

The First Hospital of Jilin University

Changchun, 130021, China

Location

West China Hospital of Sichuan University

Chengdu, 610041, China

Location

Guangzhou First Municipal People's Hospital

Guangzhou, 510180, China

Location

Hainan General Hospital

Haikou, 570311, China

Location

First Affiliated Hospital Medical School of Zhejiang University

Hangzhou, 310003, China

Location

Lanzhou University Second Hospital

Lanzhou, 730030, China

Location

Jiangsu Province Hospital

Nanjing, 210029, China

Location

Affiliated Hospital of Nantong University

Nantong, 226001, China

Location

Shanghai Zhongshan Hospital

Shanghai, 200032, China

Location

Huashan Hospital affiliated to Fudan University

Shanghai, 200040, China

Location

The First Affiliated Hospital of Soochow University

Suzhou, 215006, China

Location

Tianjin Medical University General Hospital

Tianjin, 300052, China

Location

Union Hospital Tongji Medical College of Huazhong University of Science and Technology

Wuhan, 430022, China

Location

The First Affiliated Hospital of Xian Jiaotong University

Xi'an, 710061, China

Location

Fakultni nemocnice Brno

Brno, 625 00, Czechia

Location

Fakultni nemocnice Kralovske Vinohrady

Prague, 100 34, Czechia

Location

Ustav Hematologie A Krevni Transfuze

Prague, 128 00, Czechia

Location

Mansoura University Hospital

Al Mansurah, 35516, Egypt

Location

Alexandria University Hospital

Alexandria, 21131, Egypt

Location

Cairo university

Cairo, 11211, Egypt

Location

Ain Shams University Hospital

Cairo, 1181, Egypt

Location

National Cancer Institute

Cairo, 11976, Egypt

Location

Nasser institute Hospital

Cairo, 12655, Egypt

Location

ICH Hopital A. Morvan

Brest, 29609, France

Location

CHU Dijon Bourgogne

Dijon, 21000, France

Location

CHU Grenoble

La Tronche, 38700, France

Location

CHU Nantes - Hotel Dieu

Nantes, 44093, France

Location

CHU De Poitiers

Poitiers, 86021, France

Location

CHRU Hopital Sud

Rennes, 35200, France

Location

Onkologische Schwerpunktpraxis

Berlin, 10707, Germany

Location

Universitatsklinikum Essen

Essen, 45147, Germany

Location

Rotkreuzklinikum München

München, 80634, Germany

Location

General Hospital of Athens G Gennimatas

Athens, 115 27, Greece

Location

Laiko General Hospital of Athens

Athens, 11527, Greece

Location

Attikon University General Hospital of Attica

Athens, 12462, Greece

Location

University Hospital Of Larissa

Larissa, 41110, Greece

Location

University General Hospital of Rio Patras

Pátrai, 26504, Greece

Location

University General Hospital of Thessaloniki

Thessaloniki, 54636, Greece

Location

Semmelweis Egyetem

Budapest, 1083, Hungary

Location

Del Pesti Centrumkorhaz Orszagos Hematologiai es Infektologiai Intezet Szent Laszlo Telephely

Budapest, 1097, Hungary

Location

Debreceni Egyetem Klinikai Kozpont

Debrecen, 4032, Hungary

Location

Petz Aladar Megyei Oktato Korhaz

Gyõr, 9023, Hungary

Location

Somogy Megyei Kaposi Mor Oktato Korhaz

Kaposvár, 7400, Hungary

Location

Ha'Emek Medical Center

Afula, 1834111, Israel

Location

Soroka Medical Center

Beersheba, 8410101, Israel

Location

Rambam Health Care Campus

Haifa, 3109601, Israel

Location

Shaare Zedek Medical Center

Jerusalem, 9103102, Israel

Location

Hadassah Medical Center

Jerusalem, 9112001, Israel

Location

Hospital Sanz Medical Center Laniado Hospital

Kiryat Tzanz, 4244916, Israel

Location

Rabin Medical Center, Beilinson Hospital

Petach Tikvah, 49100, Israel

Location

Tel Aviv Sourasky Medical Center

Tel Aviv, 64239, Israel

Location

PO C e G Mazzoni AST Ascoli Piceno

Ascoli Piceno, 63100, Italy

Location

ASST Spedali Civili di Brescia

Brescia, 25123, Italy

Location

Azienda Ospedaliera di Rilievo Nazionale e di Alta Specializzazione Garibaldi

Catania, 95122, Italy

Location

AOU Policlinico G Rodolico S Marco

Catania, 95125, Italy

Location

Azienda Ospedaliero Universitaria Careggi

Florence, 50134, Italy

Location

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

Location

Fondazione IRCCS San Gerardo dei Tintori

Monza, 20900, Italy

Location

Azienda Ospedaliero Universitaria Maggiore della Carita di Novara

Novara, 28100, Italy

Location

Umberto I Policlinico di Roma

Roma, 00161, Italy

Location

Fondazione Policlinico Universitario A Gemelli IRCCS

Roma, 00168, Italy

Location

Azienda Ospedaliera Universitaria Citta della Salute e della Scienza di Torino

Torino, 10126, Italy

Location

Azienda Ulss 8 Berica- Ospedale Di Vicenza

Vicenza, 36100, Italy

Location

Fukushima Medical University Hospital

Fukushima, 960 1295, Japan

Location

Saitama Medical University Hospital

Iruma-gun, 350-0495, Japan

Location

St Marianna University Hospital

Kanagawa, 216 8511, Japan

Location

Japan Community Health care Organization Kyushu Hospital

Kitakyusyu-Shi, 806-8501, Japan

Location

Yamanashi Prefectural Central Hospital

Kofu, 400-0027, Japan

Location

Ogaki Municipal Hospital

Ōgaki, 503-8502, Japan

Location

Sapporo Medical University Hospital

Sapporo, 060-8543, Japan

Location

Tohoku University Hospital

Sendai, 980 8574, Japan

Location

Osaka University Hospital

Suita, 565 0871, Japan

Location

Toyama Prefectural Central Hospital

Toyama, 930 8550, Japan

Location

Wakayama Medical University Hospital

Wakayama, 641 8510, Japan

Location

Tottori University Hospital

Yonago, 683-8504, Japan

Location

Hospital Ampang

Ampang, 68000, Malaysia

Location

Hospital Sultanah Aminah

Johor Bharu, 80100, Malaysia

Location

Hospital Queen Elizabeth

Kota Kinabalu, 88200, Malaysia

Location

University Malaya Medical Centre

Kuala Lumpur, 50603, Malaysia

Location

Hospital Tengku Ampuan Afzan

Kuantan, 25100, Malaysia

Location

Hospital Umum Sarawak

Kuching, 93586, Malaysia

Location

Sunway Medical Centre

Petaling Jaya, 47500, Malaysia

Location

Amsterdam Universitair Medische Centra Locatie Vrije Universiteit Medisch Centrum

Amsterdam, 1081 HV Amsterdam, Netherlands

Location

Academic Medical Centre Amsterdam

Amsterdam, 1105 AZ, Netherlands

Location

Maastricht Universitair Medisch Centrum

Maastricht, 6229 HX, Netherlands

Location

Interhem

Bialystok, 15-732, Poland

Location

Klinika Hematologii i Transplantologii, UCK

Gdansk, 80-214, Poland

Location

Pratia Onkologia Katowice

Katowice, 40 519, Poland

Location

Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im M Kopernika w Lodzi

Lodz, 93 510, Poland

Location

Szpital Wojewodzki w Opolu

Opole, 45-061, Poland

Location

MTZ Clinical Research Powered by Pratia

Warsaw, 02-172, Poland

Location

Kyungpook National University Hospital

Daegu, 41944, South Korea

Location

Chungnam National University Hospital

Daejeon, 35015, South Korea

Location

Samsung Medical Center

Seoul, 06351, South Korea

Location

Asan Medical Center

Seoul, 138-040, South Korea

Location

Hosp. Univ. Principe de Asturias

Alcalá de Henares, 28805, Spain

Location

Hosp. Univ. Germans Trias I Pujol

Badalona, 08916, Spain

Location

Hosp Clinic de Barcelona

Barcelona, 08036, Spain

Location

Complejo Asistencial Univ. de Burgos

Burgos, 09006, Spain

Location

Hosp Reina Sofia

Córdoba, 14004, Spain

Location

Hosp. Univ. de Gran Canaria Dr. Negrin

Las Palmas de Gran Canaria, 35010, Spain

Location

Hosp. Univ. Infanta Leonor

Madrid, 28031, Spain

Location

Hosp. Univ. Ramon Y Cajal

Madrid, 28034, Spain

Location

Hospital Clinico San Carlos

Madrid, 28040, Spain

Location

Hosp. Univ. 12 de Octubre

Madrid, 28041, Spain

Location

Hosp. Univ. La Paz

Madrid, 28046, Spain

Location

Hospital Universitario Puerta de Hierro Majadahonda

Majadahonda, 28222, Spain

Location

Hosp Regional Univ de Malaga

Málaga, 29010, Spain

Location

Hosp Virgen de La Victoria

Málaga, 29010, Spain

Location

Hosp. Virgen Del Rocio

Málaga, 29010, Spain

Location

Hosp. Son Llatzer

Palma de Mallorca, 07198, Spain

Location

Clinica Univ. de Navarra

Pamplona, 31008, Spain

Location

Hosp. Quiron Madrid Pozuelo

Pozuelo de Alarcón, 28223, Spain

Location

Hosp. Univ. I Politecni La Fe

Valencia, 46026, Spain

Location

Hosp. Univ. Miguel Servet

Zaragoza, 50009, Spain

Location

Cherkassy Regional Oncology Dispensary, Department of Hematology

Cherkassy, 18009, Ukraine

Location

Dnipropetrovsk State Medical Academy, Dnipropetrovsk City Multifield Clinical Hospital # 4

Dnipro, 49102, Ukraine

Location

Medical Center 'Ok Clinic' of LLC 'International Institute of Clinical Studies'

Kyiv, 02091, Ukraine

Location

Kyiv City Clinical Hospital #9, Department of infectious diseases

Kyiv, 04112, Ukraine

Location

Ternopil University Hospital of Ternopil Regional Council

Ternopil, 46002, Ukraine

Location

Leeds Teaching Hospitals NHS Trust

Leeds, LF9 7TF, United Kingdom

Location

Liverpool University Hospitals NHS Foundation Trust

Liverpool, L7 8XP, United Kingdom

Location

Barts Health NHS Trust

London, E1 2ES, United Kingdom

Location

Oxford University Hospitals NHS Foundation Trust

Oxford, OX3 7LE, United Kingdom

Location

University Hospitals Plymouth NHS Trust

Plymouth, PL6 8DH, United Kingdom

Location

University Hospital Southampton NHS Foundation Trust

Southampton, SO166YD, United Kingdom

Location

Related Publications (1)

• Liu AP, Cheuk DK. Disease-modifying treatments for primary autoimmune haemolytic anaemia. Cochrane Database Syst Rev. 2021 Mar 26;3(3):CD012493. doi: 10.1002/14651858.CD012493.pub2.

  PMID: 33786812 DERIVED

Related Links

• Participant recruitment poster

Study Officials

• Janssen Research & Development, LLC Clinical Trial

  Janssen Research & Development, LLC

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: September 18, 2019
• First Posted: October 8, 2019
• Study Start: August 15, 2019
• Primary Completion: February 2, 2025
• Study Completion (Estimated): April 1, 2028
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

BR (14); IT (12); CZ (3); JP (12); US (29); EG (6); PL (6); GB (6); GR (6); CN (16); FR (6); MY (7); IL (8); NL (3); ES (20); HU (5); UA (5); DE (3); KR (4)